Muscle Owl Talks About SMA Research in AVXS-101

In this podcast episode of Muscle Owl Talks from Muscle Owl, host Peter Duffy chats with his co-host Michaela, a journalist from Northern Ireland who has spinal muscular atrophy (SMA).

Doctors advise care for children with SMA Type 1 based on certain factors. Read more. 

The two talk about the advances in medicine and treatments for SMA, in particular, the success seen in children with type 1 SMA who have undergone the gene therapy AVXS-101. The clinical trials have shown SMA infants who would normally be unable to breathe unaided at eight months are able to crawl like normal babies following the treatment. Peter and Michaela talk about the possibility of the treatment being passed by the FDA and applied to other similar diseases.  Find out more about the clinical trial for AVXS-101 here.

Better understand the mechanisms of spinal muscular atrophy with this animated film.

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